Trial reportJournal of comparative effectiveness research2022
Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients.
Open access · greenAbstract readClinical Trial, Phase III
In one paragraph
Trial report in Journal of comparative effectiveness research, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 31 papers, 2 of them syntheses that pooled it.
0numbers the graph read from it
0cells of the map it votes in
31citing papers in PubMed, 2 pooled it
3.8field-weighted citation impact, top 5% of its field
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
31 citing papers in PubMed, 2 syntheses or guidelines pooled it, 60 citations in OpenAlex.
- Factors Associated with Respiratory Health and Function in Duchenne Muscular Dystrophy: A Systematic Review and Evidence Grading.Journal of neuromuscular diseases · 2024Pooled it
- Predictors of Loss of Ambulation in Duchenne Muscular Dystrophy: A Systematic Review and Meta-Analysis.Journal of neuromuscular diseases · 2024Pooled it
- Confirmatory long-term efficacy and safety results of ataluren in patients with nmDMD from Study 041, an international, randomized, double-blind, placebo-controlled, Phase III trial.Journal of comparative effectiveness research · 2025Trial
- Current and Emerging Therapeutic Strategies for the Treatment of Duchenne Muscular Dystrophy.Genes · 2026Review
- Genetic, Clinical, and Management Characteristics of Duchenne Muscular Dystrophy in Saudi Arabia.Healthcare (Basel, Switzerland) · 2026Article
- Ataluren improves hematopoietic and pancreatic disorders in Shwachman-Diamond syndrome patients: a compassionate program case-series.Nature communications · 2025Article
- Natural history of patients with nonsense mutation Duchenne muscular dystrophy treated with ataluren in Spain.Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology · 2025Article
- Lost in Transition: Challenges in the Journey from Pediatric to Adult Care for a RomanianHealthcare (Basel, Switzerland) · 2025Article
- The evolving landscape of NF gene therapy: Hurdles and opportunities.Molecular therapy. Nucleic acids · 2025Review
- A New Perspective on Drugs for Duchenne Muscular Dystrophy: Proposals for Better Respiratory Outcomes and Improved Regulatory Pathways.Paediatric drugs · 2025Review
- Genetic and clinical landscape of Duchenne muscular dystrophy in Guatemala: insights from a national study.Frontiers in genetics · 2025Article
- Safety and effectiveness of ataluren in patients with Duchenne muscular dystrophy: single-center experience from Saudi Arabia.The Journal of international medical research · 2024Article
- Suppressor tRNA in gene therapy.Science China. Life sciences · 2024Review
- Pharmacological Treatments and Therapeutic Targets in Muscle Dystrophies Generated by Alterations in Dystrophin-Associated Proteins.Medicina (Kaunas, Lithuania) · 2024Review
- Extended stop codon context predicts nonsense codon readthrough efficiency in human cells.Nature communications · 2024Article
- Article
- Healthcare Stakeholder Perspectives on a Value Assessment Approach for Duchenne Muscular Dystrophy Therapies.Journal of multidisciplinary healthcare · 2024Article
- Clinical Profile and Pulmonary Function of Pediatric Patients with Duchenne Muscular Dystrophy at a Tertiary Government Hospital.Acta medica Philippina · 2024Article
- En Route to Targeted Ribosome Editing to Replenish Skin Anchor Protein LAMB3 in Junctional Epidermolysis Bullosa.JID innovations : skin science from molecules to population health · 2024Article
- Prenatal diagnosis of 1408 foetuses at risk of DMD/BMD by MLPA and Sanger sequencing combined with STR linkage analysis.BMC medical genomics · 2023Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
15 authors at 5 institutions in 3 countries.
Craig M McDonaldDepartment of Pediatrics, University of California Davis School of Medicine, Davis, CA, USA.ORCID 0000-0002-8779-3220
Francesco MuntoniDubowitz Neuromuscular Centre & MRC Centre for Neuromuscular Diseases, University College London, Institute of Child Health & Great Ormond Street Hospital for Children Foundation Trust, London, UK.ORCID 0000-0002-9102-5232
Vinay PenematsaPTC Therapeutics, South Plainfield, NJ, USA.
Joel JiangPTC Therapeutics, South Plainfield, NJ, USA.
Allan KristensenPTC Therapeutics, South Plainfield, NJ, USA.
Francesco BibbianiPTC Therapeutics, South Plainfield, NJ, USA.
Elizabeth GoodwinPTC Therapeutics, South Plainfield, NJ, USA.
Heather Gordish-DressmanCenter for Genetic Medicine, Children's National Health System & the George Washington, Washington, DC, USA.ORCID 0000-0002-2330-5427
Lauren MorgenrothTherapeutic Research in Neuromuscular Disorders Solutions, Pittsburgh, PA, USA.
Christian WernerPTC Therapeutics Germany GmbH, Frankfurt, Germany.
James LiPTC Therapeutics, South Plainfield, NJ, USA.
Richard AblePTC Therapeutics, South Plainfield, NJ, USA.
Panayiota TrifillisPTC Therapeutics, South Plainfield, NJ, USA.
Már TuliniusDepartment of Pediatrics, Gothenburg University, Queen Silvia Children's Hospital, Gothenburg, Sweden.
Study 019 investigators
PTC Therapeutics (United States) · USDrottning Silvias barn- och ungdomssjukhus · SEGreat Ormond Street Hospital · GBChildren's National · USUniversity of California, Davis · US
Funding
WU INSTITUTE OF CLINICAL AND TRANSLATIONAL SCIENCESUL1TR002345 · NCATS · WASHINGTON UNIVERSITY · PI William G. Powderly · 2017 to 2026
$97.8MWashington University Institute of Clinical and Translational Sciences (UL1)UL1RR024992 · NCRR · WASHINGTON UNIVERSITY · PI EVANOFF, BRADLEY A · 2007 to 2011
$45.4MSUPPLEMENT FOR RENOVATION OF ANIMAL FACILITIES AT INSTITUTE OF NEUROBIOLOGYG12RR003051 · NCRR · UNIVERSITY OF PUERTO RICO MED SCIENCES · PI CONDE, JOSE G · 1986 to 2011
$28.1MTRCLU54RR026139 · NCRR · UNIVERSITY OF PUERTO RICO MED SCIENCES · PI ZORRILLA, CARMEN D · 2010 to 2011
$8.4MCTSA INFRASTRUCTURE FOR PEDIATRIC RESEARCHUL1RR031988 · NCRR · CHILDREN'S RESEARCH INSTITUTE · PI GUAY-WOODFORD, LISA MARIE · 2010 to 2011
$8.2MWellstone Muscular Dystrophy Center: Children's Nat'l Med CtrU54HD053177 · NICHD · CHILDREN'S RESEARCH INSTITUTE · PI HOFFMAN, ERIC P. · 2005 to 2009
$7.3MBiomarker discovery and validation in a Duchenne dystrophy natural history studyR01AR062380 · NIAMS · UNIVERSITY OF CALIFORNIA AT DAVIS · PI HATHOUT, YETRIB, MCDONALD, CRAIG M. · 2011 to 2014
$1.5MClinically Meaningful Outcomes for Duchenne Muscular Dystrophy Therapeutic TrialsR01AR061875 · NIAMS · UNIVERSITY OF CALIFORNIA AT DAVIS · PI MCDONALD, CRAIG M. · 2011 to 2014
$1.3MDepartment of HealthNCATS NIH HHS UL1 TR002345NCRR NIH HHS G12 RR003051NCRR NIH HHS U54 RR026139NCRR NIH HHS UL1 RR024992NCRR NIH HHS UL1 RR031988NIAMS NIH HHS R01 AR061875NIAMS NIH HHS R01 AR062380NICHD NIH HHS U54 HD053177
Abstract
PubMed holds no abstract for this paper.
Indexed as
Codon, NonsenseMuscular Dystrophy, DuchenneHumansOxadiazolesWalkingatalurenCodon, NonsenseOxadiazolesatalurendystrophinefficacyloss of ambulationnonsense mutation Duchenne muscular dystrophyrespiratory functionStudy 019
Identifiers
What OpenQuestion holds
Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390
Registered trials
None linked
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.